Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

JULY 29, 2022

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

Gene Expression 57

Gene Expression Gene Genetic Disease RNA 57

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. However, these therapies can cause severe adverse effects. We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 52

The Pharma Data

DECEMBER 27, 2020

STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52